US-based Tyra Biosciences has dosed the first paediatric participant in the dose-escalation/dose-expansion Phase II BEACH301 trial designed to assess the efficacy and safety of dabogratinib (TYRA-300), a treatment for children with achondroplasia, which is the most prevalent form of dwarfism.
The open-label, multi-centre trial targets children aged between three and ten years who have achondroplasia with open growth plates.
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It is set to include two cohorts of children: those who have not previously undergone treatment in Cohort 1 and those who have been subjected to growth-accelerating therapy in Cohort 2.
It is anticipated that up to ten subjects will be enrolled per dose level in each cohort, with the study lasting up to 12 months.
The dose levels being tested are 0.125mg/kg, 0.25mg/kg, 0.375mg/kg, and 0.50mg/kg.
Additionally, a safety sentinel cohort will be established, consisting of up to three subjects/dose level, focusing on those who are aged five to ten years.
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By GlobalDataThe primary goals of the BEACH301 trial will evaluate the tolerability and safety of the oral therapy in the paediatric population with this condition and measure the change in annualised growth velocity from baseline.
This will aid in determining the appropriate dose(s) for subsequent phases of development.
Secondary objectives of the trial include the assessment of changes from baseline in height z-score, pharmacokinetics and proportionality.
Developed using the company’s SNÅP platform, dabogratinib is a once-a-day medication that acts as a selective inhibitor of the fibroblast growth factor receptor 3 (FGFR3).
Tyra Biosciences CEO Todd Harris said: “Achondroplasia is caused by an alteration in FGFR3, and we believe that precisely targeting the root cause of this condition is the key to transforming care.
“Dabogratinib is the only oral, FGFR3-selective inhibitor in clinical development for achondroplasia, reflecting our commitment to bring forward therapies that are both innovative and accessible for children and their families.”
